Efficacy and Safety of Lenalidomide for Treatment of Autistic Spectrum Disorders

• Status: Completed
• Trial end date: 2009-12-01
• Target enrollment: 0
• Participant gender: Male

Summary

The purpose of this study is to determine if lenalidomide (Revlimid®)reduces proinflammatory cytokines including TNF-alpha and may actually alter the clinical course of autism for some children.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Sutter Medical Foundation
• Treatments: Lenalidomide; Thalidomide

Criteria

Inclusion Criteria:

• Diagnosis of autistic spectrum disorder as defined by DSM-IV criteria.

• Inflammatory CSF and serum markers with elevated level of TNF-Alfa (> 50pg/ml) or other Cytokine markers such as IL-1, IL-6 or MECP-1, or serum levels of such cytokines greater than 2X normal levels even in absence of CSF markers.

or

• Patients with interictal epiliptiform EEG changes in the absences of clinical seizures, if CSF inflammatory markers are identified.

• Patients will maintain any other baseline medications for autistic problems or EEG treatment as long as on these for prior 6-8 weeks with no dosage changes. Mentally impaired minors require a parent or legal guardian to sign the informed consent.

Exclusion Criteria:

• -Diagnosis of PPD-NOS and other autism spectrum disorders.

• Any serious medical condition, laboratory abnormality, genetic, brain, structural, or psychiatric illness that would prevent the subject from participating.

• History of neutropenia, thrombocytopenia or other types of myelosuppression or risk factors for myelosuppression.

• History or risk factors for thromboembolic events.

• Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study.

• Use of any other experimental drug or therapy within 28 days of baseline.

• Current use of steroids (e.g. dexamethasone, prednisone), anthracyclines (Doxil, Adriamycin).

• Known hypersensitivity to thalidomide.

• The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs.

• Any prior use of lenalidomide.

• Known positive for HIV or infectious hepatitis, type A, B or C or tuberculosis.